This study assessed the budget impact (BI) of introducing noninvasive perinatal testing (NIPT) to the Korean National Health Insurance (KNHI). It aims to provide useful economic evidence on reimbursement decisions on prenatal screening. Since NIPT is widely used in Korea as a noncovered service, BI can be reliably estimated from real-world data (RWD).

In this BI analysis, we derived the total number of pregnant women from 2017 birth statistics (n=358,000) and its distribution of singleton births (96.1%) and multiples (3.9%). The age distribution of pregnant women was 29.4 percent for 35 and above, and 70.6 percent for below 35, according to the 2017 statistics. For NIPT costs, we considered a price range of KRW357,000 (USD260) to KRW715,000 (USD522) from a 2018 survey. To evaluate the BI of introducing NIPT, we examined eight scenarios based on NIPT cost sharing of 30 percent and 70 percent, NIPT price, and whether to include pregnant women aged less than 35 years old.

BI for KNHI was estimated as KRW6 trillion (USD4 billion) to approximately KRW56 trillion (USD40 billion). Scenario seven, targeting older pregnant women with serum screening high risk with NIPT price at the lower end and payer coverage of 30 percent, shows the lowest annual BI of KRW6,115,813,866 (USD4,469,516); scenario two, covering all older pregnant women and younger high-risk cases with NIPT price at the higher end and payer coverage of 70 percent, exhibits the highest burden at KRW56,240,636,611 (USD41,091,993).

Our BI analysis of introducing NIPT to KNHI can serve as essential data for estimating insurer burdens during the potential transition of NIPT to provisional or selective coverage. In the absence of prenatal diagnostics cost-effectiveness research in Korea, our findings provide crucial evidence for establishing relevant reimbursement criteria, addressing a gap in current research and supporting evidence-based policymaking.

The majority of those infected with chronic hepatitis C (CHC) are asymptomatic. Population screening has proven to be both effective and cost effective. When considering whether to implement screening or not, the uncertainty of the budget impact plays an important role. This study aims to develop methods that improve the accuracy of budget impact analysis for a one-time CHC screening program.

We developed a back-calculation mathematical model that employs a Markov chain Monte Carlo algorithm to estimate the prevalence and proportion of undiagnosed CHC. Subsequently, we utilized a state-transition model to assess the budget impact of two strategies: (i) no screening; and (ii) screen-and-treat with direct-acting antiviral (DAA) for individuals born between 1945 and 1965 (“baby-boomer” birth cohort). Model data were gathered from published literature. Our analysis adopted a Canadian provincial payer perspective, employed a 10-year time horizon, and followed best-practice recommendations by not applying discounting.

For individuals born between 1945 and 1965, the estimated prevalence of CHC was 1.74 percent (95% confidence interval [CI]: 1.52, 2.30) with an undiagnosed proportion of 15.72 percent (95% CI: 11.27, 18.54). The initial budget impact analysis indicated an additional cost of CAD61.5 million (USD45.0 million) over 10 years for screening related individuals for CHC in Ontario. With these updated prevalence and undiagnosed proportion estimates, our projection suggests a 29.6 percent reduction in the budget impact, now estimated at CAD43.3 million (USD31.7 million).

By comparing the budget impact of the CHC screening strategy with other recommended health services and technologies in Ontario, we have concluded that CHC screening may be considered affordable. To enhance the accuracy of budget impact analysis for population-level screening decision-making, it is crucial to develop precise methodologies for estimating the underlying prevalence and undiagnosed proportions.

In Italy, there is a lack of evidence regarding the care and management of patients with dementia, as well as the associated costs. This study aims to fill this informational gap by utilizing data from both literature and national surveys.

A prevalence-based cost-of-illness (COI) model was developed to assess dementia-related costs from a societal perspective. The resources utilization for management and treatment of patients with dementia was derived from both the literature and the analysis of surveys conducted by the National Institute of Health on the social-health structures dedicated to dementia. Indirect costs from informal caregiving were evaluated through a human capital approach. Additionally, a cost–consequence analysis (CCA) was conducted to assess the economic impact of healthcare resource utilization changes.

Based on an estimated 1,150,691 dementia cases in Italy, with approximately 12 percent institutionalized, the COI model estimated an annual expenditure of around EUR23.6 billion (USD25.7 billion) for dementia patient management, with 63 percent attributed to out-of-pocket expenses. CCA indicated that if all affiliated with Centers for Cognitive Disorders and Dementia (CDCD) received nonpharmacological interventions (versus the surveyed 25.5 percent), there would be a direct cost increase of approximately EUR4.3 million (USD4.7 million).

This analysis provides an updated overview of current dementia patient management in Italy, offering valuable insights for decision-makers to prioritize health policies and interventions for patients and their caregivers.

Dengue, a mosquito-borne disease, is prevalent in Brazil, ranking third in the Americas. The country has confirmed all four serotypes (DENV-1, DENV-2, DENV-3, and DENV-4) of the virus. In 2023, a tetravalent vaccine, approved for individuals aged four to 60 years, opens the possibility of integration into the public health system.

The invested amount for purchasing two doses of the vaccine to immunize the entire eligible population in the year 2023 was calculated. A comprehensive analysis of direct and indirect costs was conducted regarding the integration of the tetravalent dengue virus vaccine into Brazil’s public healthcare system. Direct costs encompassed treatment expenses for mild, moderate, and severe cases, while indirect costs involved workdays lost and mortality-related expenses. Direct and indirect costs were compared in two scenarios (with and without vaccination), followed by a cost–benefit ratio calculation.

The investment for procuring vaccines for over 168 million Brazilians amounted to approximately EUR17.9 billion, resulting in savings of about EUR193.5 million. Indirect costs were particularly significant when compared to the non-vaccinated population. Considering herd immunity and reducing the vaccinated population to 70 percent of the eligible populace, the invested amount was approximately EUR12.5 billion, while savings reached EUR214.2 million. A cost-benefit ratio calculation revealed a return of one centavo (EUR0.0019) for every BRL1.00 invested (EUR0.0019), and considering herd immunity, the cost–benefit ratio was approximately 0.02.

Despite Brazil being one of the countries with the highest prevalence of dengue in the Americas, the availability of the tetravalent dengue virus vaccine in the country’s public system does not seem to be a sustainable option, given the unfavorable cost–benefit analysis for such implementation. Nevertheless, it is imperative to conduct this analysis with due consideration for alternative scenarios.

Methods and processes for health technology assessment (HTA) must be sufficiently flexible to reflect emerging best practice and adapt to changes in the health and care landscape. They must also be clearly documented and subject to review, assessment, and consultation. This requires a framework to flexibly update HTA manuals, while providing stability and predictability for stakeholders.

The National Institute for Health and Care Excellence (NICE) is introducing a modular approach to updating its manuals. Previously, NICE completed full, but infrequent, updates to its manuals. A modular update is a review of the process and/or methods informing NICE’s guidance in a specific subject area, which can then be updated in the appropriate manual(s) if required. We are developing a modular updates framework that sets out NICE’s approach for identifying and prioritizing modular updates to review, reviewing the evidence and proposing changes to the manual(s), engaging and consulting with stakeholders, and implementing the modular updates.

The proposed approach allows external stakeholders to contribute to topic identification and the content of updates, helping to ensure that the manuals meet the needs of users. The first modular update to the NICE manual for health technology evaluations was published in October 2023. This included updated processes for carrying out cost comparisons, streamlining committee decision-making and handling confidential information. Potential future modular updates may include updated methods to consider health inequalities.

The introduction of a modular approach will enable NICE to be more agile and responsive in monitoring, reviewing, and improving our methods and processes, making sure they remain cutting edge as the health and care landscape continues to evolve.

Experts emphasize the necessity of reconsidering the approaches used in the health technology assessment (HTA) of digital health technologies (DHTs) to adequately address their particular characteristics. In this context, the Agency for Health Quality and Assessment of Catalonia was commissioned to develop a specific framework for the assessment of DHTs in collaboration with the agencies that constitute the Spanish Network of Agencies for Assessing National Health System Technologies and Performance (RedETS).

To accomplish the assignment, a combination of methodologies was used. To establish the domains and dimensions, an international survey, a scoping review of the literature, and a thematic analysis were executed. To determine the evidence standards that DHTs must achieve according to the risk classification derived from their use, the updated version of the evidence standards framework (ESF) from the National Institute for Health and Care Excellence (NICE) was adapted to the Spanish context. National experts in the fields of HTA and DHTs collaborated on different tasks to develop the framework.

The framework encompasses three distinct sections. The first section provides an introduction, detailing a DHT classification system that is based on the system proposed by NICE in their ESF. Following this, Section A elaborates on a comprehensive set of 13 domains, 41 dimensions, and nine sub-dimensions, offering a detailed description of each. Section B consists of the adaptation of the 21 evidence standards from the NICE ESF to the Spanish context. Of note, a clear link was established between the classification system, the domains, dimensions, sub-dimensions, and the evidence standards.

The resulting framework was published in November 2023. Since its publication, it has become the reference framework for the HTA of DHTs within the Spanish National Health System, primarily by the HTA agencies that constitute RedETS. The document will be periodically reviewed and, if necessary, adapted to align with emerging technologies and changes in legislation.

Public healthcare institutions (PHIs) experience rising and discordant implant costs. Implementing subsidies for implants without concurrent price harmonization through national procurement contracts impedes equitable patient access. This abstract describes the collaboration between the Agency for Care Effectiveness (ACE), Singapore’s national health technology assessment (HTA) agency, and the public healthcare supply chain agency (ALPS) to establish the Implant Subsidy List (ISL).

The alignment between ISL and concurrent national procurement contracts for implants required close coordination and co-development of streamlined and scalable processes. To achieve this, ACE and ALPS collaborated to understand the complex implant landscape by leveraging on known vendors and products and pre-empting upcoming ones, identifying key opinion leaders, and sharing technical expertise. Both agencies also developed common-use templates and streamlined vendor submission processes. Of note, joint request-for-proposal (RFP) exercises for national subsidy and procurement listings combined negotiation efforts to achieve lower implant prices. Regular cross-agency discussions enabled continuous knowledge sharing and process refinement in an evolving implants landscape.

For phase one rollout of ISL, 15 implant topics across eight clinical functions were evaluated, supported by concurrent establishment of 15 national procurement contracts. As of December 2023, ACE listed 22,689 ISL implants for implementation, with more topics onboarded on a rolling basis. ACE and ALPS streamlined process flows, standardized communications channels and information, and utilized contractual tools. Vendors could submit for both subsidy and procurement through the secure ALPS portal, reducing duplication of work and errors while allowing procurement and subsidy consideration processes to proceed concurrently. Combined negotiation efforts have resulted in an average price reduction of 19 percent.

The alignment between national HTA and procurement agencies is important for successful scaling. The ACE–ALPS collaboration has synergized both agencies’ understanding of the complex implant landscape and established streamlined workplans and processes to support initial establishment of ISL and its maintenance. Challenges include navigating strict procurement rules and continuous cross-agency improvement efforts to ensure applicability in an evolving landscape.

The EU HTA member state coordination group has finalized methodological guidance on indirect comparisons that states that propensity score (PS) methods should generally be used for indirect comparisons of non-randomized data in joint clinical assessments (JCAs). Half of new oncology approvals by the European Medicines Agency (EMA) between 2020 and 2023 were based on non-randomized data. This study aimed to identify how many of these were able to submit PS-based comparisons.

Using IQVIA’s Market Access Insights (MAI) database of HTAs and regulatory approvals, we characterized evidence packages submitted to EMA and HTA agencies of EU member states according to the use of PS-based comparisons and access to individual patient data (IPD) from comparator studies.

Of the 56 oncology approvals between 2020 and 2023, 30 (54%) were based on non-randomized data, of which 23 (23%) submitted PS-based indirect comparisons to EMA (15 therapies) or to HTA agencies (23 therapies). Electronic health record (EHR) or chart reviews were the most common source of comparative RWE, but agencies only took this evidence into account in fewer than half of HTAs where it was available. Use of PS-based methods also did not lead to more positive HTA outcomes than the alternative unanchored matching-adjusted indirect comparisons (MAICs) to aggregated data.

The prevalence of oncology approvals based on single-arm trials is expected to be a key challenge to the success of JCA. Unanchored comparisons will be required, but IPD was not necessarily shown to reduce uncertainty in HTAs analyzed in this research, and in about half of cases, comparisons to aggregate data were preferred due to applicability and heterogeneity concerns. Thus, the source of comparator data appears more relevant than the comparison method in HTAs, which contrasts with the available EU HTA coordination group guidance that focuses mainly on methodological aspects.

Involving the public is essential to building trust in health technology assessment (HTA) organizations. The National Institute for Health and Care Excellence (NICE) runs a deliberative public engagement program, NICE Listens. It was used to explore informed public opinion on how environmental sustainability should be taken into account in HTA.

Twenty-three general public participants from across England took part in three iterative online workshops (each lasting two or three hours, held three weeks apart in 2022). The workshops included trade-off exercises, role-play, group discussion, and video clips from interviews with sustainable healthcare experts.

Strong support was found for NICE taking action to make healthcare more environmentally sustainable. Support increased as participants learned that sustainable healthcare offers co-benefits, such as reduced burden on the National Health Service through better self-management of conditions. Participants did not want health outcomes to be compromised in pursuit of sustainability. We identified some circumstances where they found it acceptable to consider the environmental impact of interventions in decision-making: when effective treatments already exist; when the condition is not severe; when the alternative is equally cost effective; and when greener options are marginally higher in cost but as clinically effective as the alternative.

The findings demonstrate that environmental sustainability is clearly considered a relevant element of value. They also offer insight into how the environmental impact of health interventions should be considered in HTA. Further research should focus on methods for consistent measurement of the environmental impacts of health interventions and the incorporation of those impacts into decision-making.

This study aimed to map strategies for educating laypeople about health technology assessment (HTA). Although integrating community is challenging, the engagement of patients/public in the processes of HTA has garnered support and endorsement from international network agencies. Dissemination of information, educational empowerment, and training are vital to give individuals capacity to partake in the intricate web of processes actively.

This review considered studies addressing educative strategies to train laypeople on HTA, additionally mapping and summarizing relevant methodological papers from any international HTA agency. Four databases were searched for qualitative, quantitative, and mixed methods study designs. The grey literature search included policy and practice documents from HTA and health organization websites. Two reviewers independently completed title and abstract screening before the full-text review and data extraction.

The main contributors to the production of knowledge about educating laypeople in HTA were the United Kingdom (40%), Spain (20%), and Canada (13%). Most studies included were conducted in the context of the United Kingdom (27%), followed by Spain (20%), and international networks context (20%). The main strategies included conference-like events (21%), the production of educational materials (18%), training (11%), and the use of plain language (8%). Furthermore, international HTA and health agencies have offered courses, and online training produced and made available online guidance materials for increasing laypeople’s participation in the HTA process.

Despite the global efforts to educate laypeople on HTA, jurisdictional variations underscore the need for a more inclusive approach. Strategies like events, educational material production, training, and clear-language use offer diverse avenues for public engagement. International agencies’ commitment to courses, online training, and guidance reflects a collective effort to enhance public involvement.

In 2025, oncology drugs with new active substances and advanced therapy medicinal products will undergo joint clinical assessment (JCA). The comparative analysis of the clinical evidence as defined in the Regulation (EU) 2021/2282 on health technology assessment (HTAR) will save national/regional submissions of the same evidence. JCA will be available early supporting appraisal and decision-making, which remains within the responsibility of member states (MS).

Targeted searches on JCA and statements from stakeholders were performed and analyzed. We conducted interviews with current and former national payers, as well as members of HTA agencies, across Germany, France, Italy, and Eastern Europe to explore their perspectives on the anticipated implications of JCA on decision-making processes and reimbursement strategies in Europe. Focus was on reduced/additional effort for authorities and health technology developers (HTDs), required national amendments, and potential discrepancies between JCA outcome and MS benefit evaluations.

Stakeholders appreciate the standardized methodology and guidance on HTA, which, especially in countries without an established HTA system, could enhance patients’ access to new treatments by considering JCA in decision-making. The comprehensive evidence compilation may also save resources in pursuing national/regional submissions. On the other hand, country-based appraisals within the MS could lead to diverse conclusions, and there is uncertainty as to which extent national authorities will adopt JCA and how its integration into decision-making will be handled. Some stakeholders challenge an impact on local patients’ access as reimbursement and pricing processes remain within MS responsibility.

JCA is a long-desired achievement and will set the groundwork for timely access of new treatments in the MS. However, presently there are several uncertainties on how JCA will impact decision-making and whether MS appraisal could lead to contradictory value conclusions for a given treatment. Future adjustments to national/regional procedures and refinement of the JCA framework are expected.

In Québec, Canada, decisions about implementing innovations are taken both centrally for province-wide access and locally by healthcare institutions. There is no systematic evaluation process and various stakeholders are involved, notably within a new nationwide governance structure. There was a wish to increase consistency and clarity with the principles and methods used by various bodies across the innovation lifecycle.

The starting point was the Institut national d’excellence en santé et services sociaux (INESSS) multidimensional framework, which focuses on the population-level, clinical, economic, organizational, and sociocultural value of drugs, technologies, and interventions. The framework, already under evolution drawing on Responsible Innovation in Health (RIH), evolved through collaborative work between INESSS’ methodological and scientific teams, but also and foremost with diverse groups and institutions within the provincial innovation ecosystem (e.g., university-based incubators, regional hospitals). The first steps were to capture current concepts and practices from different stakeholders, as well as their operational needs in terms of assessment tools.

This multistakeholder taskforce resulted in the development of an operational toolbox meant to guide the value appraisal of innovations through a lifecycle approach. First aimed at stakeholders involved locally in healthcare institutions, the work conducted was equally beneficial to INESSS by enabling its evaluation teams to contribute to the operational tools needed to enhance clarity and legibility of the agency’s processes and methods. The level of collaboration with stakeholders across the province was also unique and has strengthened the understandability and actionability of the toolbox developed. Some challenges were faced, and related actions will be discussed.

Both the taskforce process and its output contributed to improving consistency in the assessment of innovations across the province. They made more explicit what may sometimes be perceived as the HTA “black box.” The INESSS value appraisal framework also evolved considering key elements of responsibility from RIH and through this collaboration with stakeholders, and its applicability in different contexts was reinforced.

Appraisal decisions on additional benefit of new medicines within the German health technology assessment (HTA) body Gemeinsamer Bundesausschuss (G-BA) are made by voting among the member of the G-BA plenary. We identified and analyzed key characteristics of decisions that were not reached by consensus.

G-BA’s homepage was used to identify AMNOG (German Medicines Market Reorganization Act) procedures that started after January 2011 and were finalized before November 2023. Appraisal voting is conducted publicly, and results are documented in the data source of the German Association of Research-Based Pharmaceutical Companies (vfa). Both the payer (National Association of Statutory Health Insurance [GKV-SV]) and provider (National Association of Statutory Health Insurance Physicians, Dentists and the German Hospital Federation) “benches” have an equal number of votes with the independent chair of the G-BA acting as swing vote in case of discrepant decisions. Discrepant voting instances were extracted and analyzed.

From January 2011 to November 2023, G-BA conducted 908 appraisals of medicines. In 66 appraisals, (7.3%) decisions were not reached by consensus. Discrepant voting was related to oncological (n=28), metabolic (n=15), infectious (n=12), neurologic (n=3), cardiovascular (n=2), psychiatric (n=2), dermatologic (n=2), musculoskeletal (n=1) and urogenital conditions (n=1) conditions. Fourteen discrepant voting instances related to orphan medicines. The best benefit category reached in the 66 discrepant decisions were: major (n=2), considerable (n=16), minor (n=19), non-quantifiable (n=13), and no benefit (n=16). In all discrepant voting decisions, the provider bench favored a better scoring versus the payer bench.

Appraisal decisions within G-BA are reached by voting. The appraisals are a key element within the subsequent price negotiations. In all discrepant decisions, the payer bench suggests less benefit (strength of benefit, respectively) versus the provider bench, indicating a procedural challenge with the GKV-SV being involved in both the voting on the additional benefit and the negotiation of price.

Health technology assessment (HTA) plays a pivotal role in healthcare decision-making, evaluating the cost-effectiveness of emerging technologies. Central and Eastern Europe (CEE) presents a unique context for HTA, marked by diverse healthcare systems, economic variations, and regulatory frameworks. This study addresses the critical issue of transferability of economic models within HTA in CEE, aiming to bridge existing gaps and enhance the region’s capacity for informed decision-making.

A comprehensive and systematic approach was employed to assess the transferability of economic models in the context of HTA across CEE. We conducted an extensive literature review, analyzed HTA reports, and engaged in expert consultations to understand the nuances of the healthcare systems in the region. Key factors influencing the transferability of economic models, such as healthcare infrastructure, economic disparities, and regulatory landscapes, were systematically evaluated. The study focused on a range of economic models commonly used in HTA, including cost-effectiveness analysis, budget impact analysis, and multiple-criteria decision analysis.

Our findings highlight the intricate dynamics influencing the transferability of economic models within HTA in CEE. While certain economic models exhibit a degree of generalizability across the region, there are notable variations based on specific contextual factors. Economic models designed for Western healthcare systems may not seamlessly translate to the CEE context due to differences in healthcare delivery, patient populations, and policy frameworks. The study identifies critical determinants of transferability, including the level of healthcare infrastructure development, economic disparities among CEE countries, and the diversity in regulatory approaches.

In conclusion, this study emphasizes the need for a nuanced and context-specific approach to the transferability of economic models within HTA in CEE. Bridging the gap in transferability enhances the region’s capacity for evidence-based healthcare resource allocation and contributes to the overall efficiency and sustainability of healthcare systems in CEE.

The adoption and development of health technology assessment (HTA) in the hospital setting (HB-HTA) differs among countries, but in all cases, it encounters barriers and facilitators. An analysis was conducted to promote international cooperation to develop strategies both to enforce common facilitators and overcome common barriers. HTA specialists from seven countries (at least two per country) contribute to the study.

HTA experts from countries in Western Europe (three), Central and Eastern Europe (three), and Central Asia (one) voluntarily participated in the project. They provided a description of the scenario that HB-HTA faces in their country, then a two-round Delphi study was conducted. The survey was based on twelve statements that were categorized into external and internal barriers and external and internal facilitators. Next, panel experts ranked statements on a seven-point Likert scale with a median agreement score greater than or equal to six and an interquartile range (IQR) greater than or equal to one accepted as reaching consensus. The goal was to identify similarities and differences in the HB-HTA scenarios among countries.

Fifteen experts from France, Hungary, Italy, Kazakhstan, Poland, Switzerland, and Ukraine contributed to the analysis. Among the twelve statements, six were ranked as reaching consensus (two barriers, four facilitators). One external and one internal barrier, which reached consensus, were (i) lack of formal recognition of the role of HB-HTA in national/regional legislations and (ii) limited human resources. Two external facilitators were (iii) creation of a network among hospitals performing HB-HTA and (iv) dissemination of HB-HTA methods and activities, while two internal facilitators were (v) top hospital management support in evidence-based decision-making and (vi) training initiatives dedicated to HB-HTA.

The analysis showed a consensus on the barriers and facilitators for HB-HTA. This creates the opportunity for internationally developed strategies to enforce facilitators and support the adoption and sustainability of HB-HTA. Future research should extend the analysis to other countries and include the results of the HB-HTA survey conducted in 2023 by the HB-HTA Interest Group.

We compared the Institute for Clinical and Economic Review’s (ICER) ratings of comparative clinical effectiveness with the German Federal Joint Committee’s (G-BA) added benefit ratings, and explored what factors, including the evidence base, may explain disagreement between the two organizations.

Drugs were included if they were assessed by ICER under its 2020–2023 Value Assessment Framework and had a corresponding assessment by G-BA as of March 2023 for the same indication, patient population, and comparator drug. To compare assessments, we modified ICER’s proposed crosswalk between G-BA and ICER benefit ratings to account for G-BA’s extent and certainty ratings. We also determined whether each assessment pair was based on similar or dissimilar evidence. Assessment pairs exhibiting disagreement based on the modified crosswalk despite a similar evidence base were qualitatively analyzed to identify reasons for disagreement.

We identified 15 assessment pairs and seven out of fifteen were based on similar evidence. G-BA and ICER assessments disagreed for each of these drugs. For 4/7 drugs, G-BA (but not ICER) determined the evidence was unsuitable for assessment: for 2/4 drugs, G-BA concluded the key trials did not appropriately assess the comparator therapy; for 1/4, G-BA did not accept results of a before-and-after study due to non-comparable study settings; for 1/4, G-BA determined follow-up in the key trial was too short. Among assessment pairs where both organizations assessed the evidence, reasons for disagreement included concerns about long-term safety, generalizability, and study design.

This study underscores the role of value judgments within assessments of clinical effectiveness. These judgments are not always transparently presented in assessment summaries. The lack of clarity regarding these value-based decisions underscores the need for improvements in transparency and communication, which are essential for promoting a more robust health technology assessment process and supporting transferability of assessments across jurisdictions.

The COVID-19 pandemic put substantial strain on healthcare systems globally. Early decision-making about diagnostic tests and treatments was driven by the need for rapid responses with a focus on reducing clinical burden. As COVID-19 continues its transition into an endemic state, health technology assessment (HTA) agencies will need to consider the clinical- and cost-effectiveness of tests and treatments, as with other conditions.

We first conducted a systematic literature review in July 2021 and updated the search in July 2023. The review aimed to identify economic evaluations of diagnostics for SARS-CoV-2 and treatments for COVID-19 using predefined search strategy across journal databases and sources of grey literature. In the update, an additional targeted search was completed with terms relating to novel treatments. Search results were screened by title and abstract, and full texts of potentially relevant studies were reviewed against selection criteria. Studies with very serious methodological limitations were excluded. Findings from studies were synthesized narratively due to high levels of heterogeneity.

The database search identified 8,287 unique records, of which 54 full texts were reviewed, 28 were quality assessed, and 15 were included. Three further studies were included through HTA sources and citation checking. Of the 18 studies ultimately included, 16 evaluated pharmacological treatments including corticosteroids, antivirals, and immunotherapies. Two studies in lower-income settings evaluated the cost-effectiveness of rapid antigen tests and critical care provision. In most studies, a healthcare or payer perspective was used, and the comparator was standard care. There were 17 modeling analyses and one trial-based evaluation. Cost–utility analyses using QALYs were the most common analysis type.

This update indicates that there are cost-effective treatments for COVID-19, with repurposed pharmacological treatments like dexamethasone presenting best value. There also appear to be promising options for people with severe disease alongside standard care. Future economic evaluations would benefit from reflecting the changing context around COVID-19 with parameters that reflect current circumstances, and fully incremental analyses comparing different treatment options.

Compared to single-gene BRAF testing to guide targeted treatment with BRAF/MEK inhibitors for advanced melanoma, multigene panels can identify additional gene mutations with known therapeutic or prognostic relevance. Implementation of multigene panels remains uneven across healthcare systems given an uncertain clinical and economic evidence base. We determined the population-level cost-effectiveness of multigene panels compared to single-gene BRAF testing for advanced melanoma.

Our population-based retrospective study emulated a hypothetical pragmatic trial comparing multigene panel sequencing to single-gene BRAF testing. We drew on comprehensive patient-level clinical and health administrative data between September 2016 and December 2018 in British Columbia, Canada. To emulate random treatment assignment, we 1:1 matched multigene panel patients to contemporaneous single-gene tested controls using genetic algorithm-based matching. We estimated three-year overall survival and healthcare costs (2021 CAD), and incremental net monetary benefit (INMB) for life years gained (LYG) using inverse probability of censoring weighted linear regression and nonparametric bootstrapping. We also estimated overall survival using Weibull regression and Kaplan–Meier survival analysis.

We matched 147 patients with advanced melanoma receiving multigene panel sequencing to contemporaneous single-gene-tested controls, achieving good balance for all 15 baseline clinical and sociodemographic covariates. After matching, mean incremental costs were CAD19,447 (USD14,217) (95% confidence interval [CI]: −CAD18,517 [−USD13,537], CAD76,006 [USD55,565]; p=0.41) and mean incremental LYG were 0.22 (95% CI: −0.05, 0.49; p=0.12). We found uncertain differences on overall survival using Kaplan–Meier (stratified Log-rank test p=0.11) and Weibull regression (HR: 0.73 [95% CI: 0.51, 1.03]; p=0.07) survival analysis. Cost differences were driven by systemic therapy (∆C: CAD8,665 [USD6,334]; 95% CI: −CAD36,387 [−USD26,600], CAD53,716 [USD39,268]; p=0.71). The INMB at CAD100,000(USD73,104)/LYG was CAD2,646 (USD1,934) (95% CI: −CAD30,044 [−USD21,963], CAD43,416 [USD31,739]; p=0.89), with a 52.8 percent probability of being cost effective.

There were clinically relevant but uncertain differences in improved survival associated with multigene panel sequencing for advanced melanoma, and the cost-effectiveness of panel-based testing was finely balanced. This real-world evidence generated using randomized trial design principles can support jurisdictions’ deliberations on the reimbursement of precision oncology interventions.

Allocating financial resources efficiently poses a significant challenge for health systems worldwide. In this context, economic evaluations (EE) are pivotal for the health technology assessment (HTA) process, particularly in standardizing approaches to enhance decision-making accuracy. This study focuses on delineating methodological nuances of EE submissions within Brazil’s Unified Health System (SUS).

This meta-epidemiological study analyzed dossiers submitted to the National Committee for Health Technology Incorporation (Conitec) for drug reimbursement from January 2022 to May 2023. We selected dossiers that included complete EE studies such as cost–utility or cost-effectiveness analyses. Our evaluation involved extracting data on the characteristics of the studies, reimbursement decisions, elements of the base case scenario, and parameters used in utility and sensitivity analyses. [Protocol: DOI 10.17605/OSF.IO/JVYEC]

We included 56 dossiers, leading to 11 favorable reimbursement decisions. EE study methods were cost-effectiveness (17) and cost–utility analyses (17), with some employing both (16). Markov chain models were used in 27 dossiers, primarily utilizing quality-adjusted life years as the health outcome measure. Additional outcomes included life years gained and frequency of avoided events. While utility assessment was reported in 33 dossiers, only six adjusted for age and seven accounted for disutility from adverse events. Thirty-two conducted deterministic or probabilistic sensitivity analyses, but only four presented parameters like credibility intervals, and 17 presented acceptability curves.

This study highlights the need for more standardized and refined methods in the EE submitted to Conitec. The existing EE methodological guidelines, dating from 2014, are currently under revision. This update is crucial to integrate recent advancements in health technology assessment and to better address contemporary requirements, particularly in light of the newly defined willingness-to-pay threshold.

Advances in next-generation sequencing/comprehensive genomic profiling (NGS/CGP) diagnostics require a value framework (VF) for accurate assessment within cancer care in Europe. Building on a previously established VF for diagnostic technologies in Latin America by the Institute for Clinical Effectiveness and Health Policy (IECS), this study synthesizes insights from a systematic literature review and stakeholder perspectives to inform the co-creation of an NGS/CGP diagnostic framework for healthcare decision-making.

The study utilized a mixed methods approach including a systematic literature review (SLR) and web-Delphi panel. The SLR identified and mapped existing VFs against the IECS VF, extracting non-overlapping criteria relevant to NGS/CGP in Europe. A Delphi panel further adapted and validated the framework, ensuring comprehensive stakeholder contribution and engagement. The Delphi´s first round was qualitative and involved open-ended feedback from participants to adapt the indicators and domains. Rounds two through four involved Likert-scale judgments of importance of each indicator. In rounds three and four, participants were shown the distribution of responses across stakeholders and could reconsider their answers.

The SLR revealed 42 VFs with an 83 percent criterion overlap with the IECS VF, resulting in 46 indicators forming the literature-adapted framework. Thirty-four participants completed the Delphi. In round one, 14 indicators and 22 descriptions were adapted, 11 indicators were merged, 14 were deleted, and one was kept the same resulting in 29 indicators for scoring in round two. The final VF has 27 indicators: 23 essential and four complementary; two indicators did not reach consensus. The domains included clinical impact, test performance, scientific evidence quality, non-clinical impact, health system integration, economic aspects, ethical/governance concerns, and health system priorities.

This approach has yielded a robust, stakeholder co-created VF for NGS/CGP diagnostics in oncology, tailored to the European setting. It offers a comprehensive set of criteria that extends beyond traditional health technology assessments, incorporating novel aspects like post-test data governance. This framework sets the stage for improving patient access to high-value technologies by aligning European stakeholder values across health systems.